An Orai1 gain-of-function tubular aggregate myopathy mouse model phenocopies key features of the human disease.

Calcium Signaling Mitochondria Muscle Disease ORAI1 Proteomics

Journal

The EMBO journal
ISSN: 1460-2075
Titre abrégé: EMBO J
Pays: England
ID NLM: 8208664

Informations de publication

Date de publication:
17 Oct 2024
Historique:
received: 24 01 2024
accepted: 27 09 2024
revised: 19 09 2024
medline: 18 10 2024
pubmed: 18 10 2024
entrez: 17 10 2024
Statut: aheadofprint

Résumé

Tubular aggregate myopathy (TAM) is a heritable myopathy primarily characterized by progressive muscle weakness, elevated levels of creatine kinase (CK), hypocalcemia, exercise intolerance, and the presence of tubular aggregates (TAs). Here, we generated a knock-in mouse model based on a human gain-of-function mutation which results in a severe, early-onset form of TAM, by inducing a glycine-to-serine point mutation in the ORAI1 pore (Orai1

Identifiants

pubmed: 39420094
doi: 10.1038/s44318-024-00273-4
pii: 10.1038/s44318-024-00273-4
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NIAMS NIH HHS
ID : R21 AR081068
Pays : United States
Organisme : Muscular Dystrophy Association (MDA)
ID : MDA 956014
Organisme : Fondazione Telethon (FT)
ID : GGP19231

Informations de copyright

© 2024. The Author(s).

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Auteurs

Nan Zhao (N)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Antonio Michelucci (A)

Department of Chemistry, Biology, and Biotechnology, University of Perugia, Perugia, Italy.

Laura Pietrangelo (L)

CAST, Center for Advanced Studies and Technology & DMSI, Department of Medicine and Aging Sciences, University Gabriele d'Annunzio of Chieti-Pescara, Chieti, Italy.

Sundeep Malik (S)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Linda Groom (L)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Jennifer Leigh (J)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Thomas N O'Connor (TN)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Takahiro Takano (T)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.

Paul D Kingsley (PD)

Department of Pediatrics, University of Rochester Medical Center, Rochester, NY, USA.

James Palis (J)

Department of Pediatrics, University of Rochester Medical Center, Rochester, NY, USA.

Simona Boncompagni (S)

CAST, Center for Advanced Studies and Technology & DNICS, Department of Neuroscience and Clinical Sciences, University Gabriele d'Annunzio of Chieti-Pescara, Chieti, Italy.

Feliciano Protasi (F)

CAST, Center for Advanced Studies and Technology & DMSI, Department of Medicine and Aging Sciences, University Gabriele d'Annunzio of Chieti-Pescara, Chieti, Italy.

Robert T Dirksen (RT)

Department of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA. Robert_Dirksen@URMC.rochester.edu.

Classifications MeSH